Intra-marrow transplantation of Mesenchymal Stromal Cells to treat MyeloDysplastic Syndromes: A feasibility study

Recruitment & Eligibility

Status: Withdrawn

Sex: Not specified

Target Recruitment: 29

Inclusion Criteria

* Subjects with a cytopathologically confirmed diagnosis of
(a) Patients with MDS RCUD, MDS RARS, MDS RCMD, MDS RAEB-1 and an IPSS * 1 (appendix C)
(b) An indication for treatment: patients should have had at least one red blood cell (RBC) transfusion in the two months prior to inclusion due to a hemoglobin level < 6 mmol/l,
Note: Subjects with thrombocytopenia or neutropenia are eligible.
* MDS RCUD, MDS RARS, MDS RCMD, MDS RAEB-1 and an IPSS * 1 and erythropoietin level <500 iU/l and progressive after first line erythropoietin treatment.
* MDS RCUD, MDS RARS, MDS RCMD, MDS RAEB-1 and an IPSS * 1 and 5q- cytogenetic abnormality and progressive after first line lenalidomide treatment.
* WHO performance status of 0, 1 or 2 (see appendix F)
* Written informed consent

Exclusion Criteria

* MDS RCUD, MDS RARS, MDS RCMD, MDS RAEB-1 and an IPSS * 1 and erythropoietin level <500 iU/l and not having received erythropoietin treatment
* MDS RCUD, MDS RARS, MDS RCMD, MDS RAEB-1 and an IPSS * 1 and 5q- cytogenetic abnormality and not having received lenalidomide treatment
* MDS RCUD, MDS RARS, MDS RCMD, MDS RAEB-1 and an IPSS * 1 who have been treated with immune suppressive drugs (e.g. corticosteroids, calcineurin inhibitors) during the last 6 months
* Impaired hepatic or renal function as defined by:
* ALT and/or AST > 2.5 x normal value
* Bilirubin > 2 x normal value
* Serum creatinin > 2 x normal value (after adequate hydration)
* Concurrent severe and/or uncontrolled medical condition (e.g. uncontrolled diabetes, infection, hypertension, cancer, etc.)
* Cardiac dysfunction as defined by:
* Myocardial infarction within the last 6 months of study entry, or
* Reduced left ventricular function with an ejection fraction <50% as measured by MUGA scan or echocardiogram (another method for measuring cardiac function is acceptable)
* Unstable angina
* Unstable cardiac arrhythmias

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>* The safety of intra-marrow transplantation of MSCs in low and int-1 risk IPSS<br /><br>MDS patients. </p><br>

Secondary Outcome Measures

Name	Time	Method
<p>* Efficacy (CR, PR, Hematological Improvement, SD, PD) 3 months after<br /><br>intra-marrow MSC infusion<br /><br>* Efficacy (CR, PR, Hematological Improvement, SD, PD) 12 months after<br /><br>intra-marrow MSC infusion<br /><br>* Overall survival measured from the date of first MSC infusion<br /><br>* Probability of progression to AML after intra-marrow MSC infusion<br /><br>* Number and duration of hospitalization as well as transfusion requirements<br /><br>(red cell and platelet transfusion).<br /><br>* Cytogenetic responses in CD34+ and CD45- cell fractions determined by FISH<br /><br>analyses<br /><br>* Determination of prognostic factors on response.<br /><br>* Adverse events </p><br>